Retrospective French nationwide survey of childhood aggressive vascular anomalies of bone, 1988-2009.
Fiche publication
Date publication
janvier 2010
Auteurs
Membres identifiés du Cancéropôle Est :
Pr CHASTAGNER Pascal
Tous les auteurs :
Heritier S, Le Merrer M, Jaubert F, Bigorre M, Gillibert-Yvert M, de Courtivron B, Ziade M, Bertrand Y, Carrie C, Chastagner P, Bost-Bru C, Leonard JC, Ouache M, Boccon-Gibod L, Mary P, de Blic J, Pin I, Wendling D, Revillon Y, Houdoin V, Forin V, Lepointe HD, Languepin J, Wagnon J, Epaud R, Fauroux B, Donadieu J
Lien Pubmed
Résumé
OBJECTIVE: To document the epidemiological, clinical, histological and radiological characteristics of aggressive vascular abnormalities of bone in children. STUDY DESIGN: Correspondents of the French Society of Childhood Malignancies were asked to notify all cases of aggressive vascular abnormalities of bone diagnosed between January 1988 and September 2009. RESULTS: 21 cases were identified; 62% of the patients were boys. No familial cases were observed, and the disease appeared to be sporadic. Mean age at diagnosis was 8.0 years [0.8-16.9 years]. Median follow-up was 3 years [0.3-17 years]. The main presenting signs were bone fracture (n = 4) and respiratory distress (n = 7), but more indolent onset was observed in 8 cases. Lung involvement, with lymphangiectasies and pleural effusion, was the most frequent form of extraosseous involvement (10/21). Bisphosphonates, alpha interferon and radiotherapy were used as potentially curative treatments. High-dose radiotherapy appeared to be effective on pleural effusion but caused major late sequelae, whereas antiangiogenic drugs like alpha interferon and zoledrenate have had a limited impact on the course of pulmonary complications. The impact of bisphosphonates and alpha interferon on bone lesions was also difficult to assess, owing to insufficient follow-up in most cases, but it was occasionally positive. Six deaths were observed and the overall 10-year mortality rate was about 30%. The prognosis depended mainly on pulmonary and spinal complications. CONCLUSION: Aggressive vascular abnormalities of bone are extremely rare in childhood but are lifethreatening. The impact of anti-angiogenic drugs on pulmonary complications seems to be limited, but they may improve bone lesions.
Référence
Orphanet J Rare Dis. 2010 Feb 3;5:3.